2 recruiting, 1 completed. 1 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Recruiting 1 |
| Phase 3 | 1 | Completed 1 |
Follow Arginase 1 Deficiency: new trials by email Literature Review of Arginase 1 Deficiency trials Research Report on Arginase 1 Deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD KRRO-121, Placebo - Urea Cycle Disorders, Inborn, Urea Cycle Disorders | Phase 1 / Phase 2 | Recruiting | Korro Bio, Inc. | 30 | 2026-09-28 | 2026-10-08 |
| Pegzilarginase in Subjects <24 Months Old With Arginase 1 Deficiency Pegzilarginase - Arginase 1 Deficiency | Phase 3 | Completed | Immedica Pharma AB | 3 | 2024-08-30 | 2026-05-22 |
| Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford Rare Disorders, Undiagnosed Disorders | Recruiting | Sanford Health | 20000 | 2010-07-01 | 2025-05-29 |
Hyperargininemia (3) · 3-Methylglutaconic Aciduria, Type V (1) · Achalasia Addisonianism Alacrimia syndrome (1) · Acrodysostosis (1) · Addison Disease (1) · Adult-onset citrullinemia type 2 (1) · Alagille Syndrome (1) · Alstrom Syndrome (1) · Amnesia, Transient Global (1) · Aniridia (1) · Aniridia and Absent Patella (1) · Aniridia, Partial, with Unilateral Renal Agenesis and Psychomotor Retardation (1) · Argininosuccinic Aciduria (1) · Ataxia (1) · Ataxia Neuropathy Spectrum (1)
Amino Acid Metabolism, Inborn Errors (3) · Brain Diseases (3) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Central Nervous System Diseases (3) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (3) · Genetic Diseases, Inborn (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · Nervous System Diseases (3) · Nutritional and Metabolic Diseases (3) · Genetic Diseases, X-Linked (2) · Urea Cycle Disorders, Inborn (2) · Abnormalities, Multiple (1) · Adrenal Gland Diseases (1)
Immedica Pharma AB (1) · Korro Bio, Inc. (1) · Sanford Health (1)
Australia (2) · Austria (1) · Portugal (1) · United Kingdom (1) · United States (1)